Lymphangioleiomyomatosis (LAM) Clinical Trials

Lymphangioleiomyomatosis results from uncontrolled proliferation of LAM cells, which are smooth muscle-like cells of unknown origin (possibly derived from perivascular epithelioid cells, PEComas). TSC-associated LAM results from somatic TSC1 or TSC2 mutations in LAM cells leading to constitutive mTOR activation. Sporadic LAM has somatic TSC2 mutations in LAM cells without germline TSC mutations.

The most common symptoms of Lymphangioleiomyomatosis include Progressive dyspnea on exertion, Chronic cough, usually nonproductive, Chest pain or discomfort, Hemoptysis from cyst rupture, Spontaneous pneumothorax, among others. Approximately 1 per 400,000 women; 30-40% of women with TSC develop LAM. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Lymphangioleiomyomatosis from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Research in Lymphangioleiomyomatosis centers on mTOR inhibitors that slow disease progression by suppressing the aberrant cell growth driving LAM. Sirolimus remains a well-studied mTOR inhibitor, while newer agents and combination approaches including dual mTOR/AMPK activators are in development, offering potential for even better outcomes.

Yes. As of October 21, 2026, 1 FDA-approved medicine has a current label that names Lymphangioleiomyomatosis: Sirolimus. Corticosteroids and immune globulins count once each.

Lymphangioleiomyomatosis arises from acquired (somatic) genetic changes in the affected cells, commonly involving TSC2 and TSC1, rather than from an inherited variant. Sporadic LAM arises from somatic TSC2 (less often TSC1) mutations and is not inherited; in TSC-associated LAM the germline TSC1/TSC2 variant is autosomal dominant. Approximately 1 per 400,000 women; 30-40% of women with TSC develop LAM.

For Lymphangioleiomyomatosis the genetic testing that matters is usually of the tumor or affected blood cells rather than inherited DNA, looking for TSC2 and TSC1. Sporadic LAM arises from somatic TSC2 (less often TSC1) mutations and is not inherited; in TSC-associated LAM the germline TSC1/TSC2 variant is autosomal dominant.

Those results guide treatment choice and are often the entry requirement for targeted-therapy trials, so ask whether the biopsy was sent for molecular testing and request the report. The Genetic Testing section of this page lists laboratories in the NIH Genetic Testing Registry.

Eligibility for Lymphangioleiomyomatosis clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Lymphangioleiomyomatosis clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.